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Beam Therapeutics Inc. (BEAM) received a boost from William Blair, which reacted positively to the company’s update on its alignment with the U.S. FDA, citing improved visibility into the data required for regulatory submissions. The firm pointed to progress in Beam’s sickle cell therapy risto-cel and its liver-targeted program BEAM-302.
William Blair also noted that both programs’ acceptance into the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot program could speed development and regulatory review. The brokerage firm reiterated its ‘Outperform’ rating.
Beam’s update outlined progress across its genetic disease and hematology franchises. The company said it has aligned with the FDA on a potential accelerated approval pathway for BEAM-302 in alpha-1 antitrypsin deficiency (AATD), based on biomarker endpoints. BEAM-302 has shown an in vivo correction of a disease-causing mutation in AATD, and Beam Therapeutics plans to expand patient enrollment to support a future biologics license application (BLA).
Beam Therapeutics’ stock gained over 28% On Monday afternoon.
In hematology, Beam expects to submit a BLA for risto-cel by year-end 2026. The BEACON Phase 1/2 trial is fully enrolled, manufacturing is complete, and recent data continue to show strong efficacy and a predictable manufacturing profile, potentially improving patient experience and treatment center capacity, the company said.
Beyond its clinical progress, Beam ended 2025 with an estimated $1.25 billion in cash, extending its operating runway into 2029. This positions the company to fund the anticipated launch of risto-cel and execute pivotal development plans for BEAM-302 without near-term financing risk.
Management will further discuss these developments at the J.P. Morgan Healthcare Conference on January 13.
Retail sentiment on Stocktwits remained in the ‘extremely bullish’ zone over the past 24 hours, accompanied by ‘extremely high’ message volumes.

One user expects a buyout.
Another user expects ‘good times’ ahead.
Over the past year, the stock has gained around 38%.
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